Lifelines for Health (vol 7): Medical Cannabis, The Safer Solution to Chronic Pain? by CHES

Spring 2016 I Volume 7

A CHES Publication

Actual FEIBA patient.

Indications for FEIBA [Anti-Inhibitor Coagulant Complex]

FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for: • Control and prevention of bleeding episodes • Use around the time of surgery • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes. FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation factor VIII or coagulation factor IX.

Detailed Important Risk Information for FEIBA WARNING: EVENTS INVOLVING CLOTS THAT BLOCK BLOOD VESSELS

• Blood clots that block blood vessels and their effects have been reported during postmarketing surveillance following infusion of FEIBA, particularly following the administration of high doses and/or in patients with a risk of forming blood clots. • If you experience any of these side effects, call your doctor right away. You should not use FEIBA if: • You had a previous severe allergic reaction to the product (reactions causing discomforts that are damaging and life threatening) • You have signs of development of small blood vessel clots throughout the body • You have sudden blood vessel clots or blocked blood vessels, (e.g., heart attack or stroke) Events involving blood clots blocking blood vessels can occur with FEIBA, particularly after receiving high doses and/or in patients with risk factors for clotting. Infusion of FEIBA should not exceed a dose of 100 units per kg body weight every 6 hours and daily doses of 200 units per kg of body weight. Maximum injection or infusion rate must not exceed 2 units per kg of body weight per minute.

LIVE IN THE BLEED-FREE MOMENT …with FEIBA prophylaxis patients can have more bleed-free days as compared to on-demand treatment.

Every joint bleed has the potential to do permanent damage1,2 Median ABR with prophylaxis vs on-demand3

REDUCTION in median ABR with prophylaxis treatment3

28.7 median ABR with on-demand treatment3,4 629 bleeding episodes occurred during on-demand treatment3,4

7.9 median ABR with

prophylaxis treatment3,4

NO BLEEDS occurred in 18% (3 out of 17) of patients on FEIBA prophylaxis in a clinical study †3

196 bleeding episodes occurred during prophylaxis treatment3,4

*Based on the results from the FEIBA PROOF clinical study of 36 hemophilia A and B patients with inhibitors receiving FEIBA for prophylaxis or on-demand treatment for 12 months.4 †Of those patients who achieved zero bleeding events, two-thirds completed 12 months of the study.4

FEIBA is the ONLY FDA-approved treatment indicated for use in hemophilia A or B patients with inhibitors for routine prophylaxis.3

Detailed Important Risk Information For FEIBA Continued At first sign or symptom of a sudden blood vessel clot or blocked blood vessel (e.g., chest pain or pressure, shortness of breath, altered consciousness, vision, or speech, limb or abdomen swelling and/or pain), stop FEIBA administration promptly and seek emergency medical treatment. Allergic-type hypersensitivity reactions, including severe, sometimes fatal allergic reactions that can involve the whole body, can occur following the infusion of FEIBA. Call your doctor or get emergency treatment right away if you get a rash, hives or welts, experience itching, tightness of the throat, vomiting, abdominal pain, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting. Because FEIBA is made from human plasma it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. The most frequent side effects observed during the prophylaxis trial were anemia, diarrhea, bleeding into a joint, signs of hepatitis B surface antibodies, nausea, and vomiting. The serious side effects seen with FEIBA are allergic reactions and clotting events involving blockage of blood vessels, which include stroke, blockage of the main blood vessel to the lung, and deep vein blood clots. Call your doctor right away about any side effects that bother you during or after you stop taking FEIBA. Please see next page for FEIBA Brief Summary of Prescribing Information. To see the Full Prescribing Information, including Boxed Warning on blood clots, go to www.FEIBA.com. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. References: 1. Pergantou H, Matsinos G, Papadopoulos A, Platokouki H, Aronis S. Comparative study of validity of clinical, X-ray and magnetic resonance imaging scores in evaluation and management of haemophilic arthropathy in children. Haemophilia. May 2006;12(3):241-247.2. Gringeri A, Ewenstein B, Reininger A. The burden of bleeding in haemophilia: is one bleed too many? Haemophilia. Jul 2014;20(4):459-463.3. FEIBA Prescribing Information. Westlake Village, CA: Baxter Healthcare Corporation; November 2013. 4. Antunes SV, Tangada S, Stasyshyn O, et al. Randomized comparison of prophylaxis and ondemand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors. Haemophilia. 2014;20(1):65-72. Baxalta and Feiba are trademarks of Baxalta Incorporated April 2016 USBS/145/16-0015

FEIBA [Anti-Inhibitor Coagulant Complex] For Intravenous Use, Lyophilized Powder for Solution Brief Summary of Prescribing Information. Please see package insert for full Prescribing Information. WARNING: THROMBOEMBOLIC EVENTS • Thromboembolic events have been reported during post-marketing surveillance following infusion of FEIBA, particularly following the administration of high doses and/or in patients with thrombotic risk factors. • Monitor patients receiving FEIBA for signs and symptoms of thromboembolic events. INDICATIONS AND USAGE FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for: • Control and prevention of bleeding episodes • Perioperative management • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes. FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation factor VIII or coagulation factor IX. CONTRAINDICATIONS • Known anaphylactic or severe hypersensitivity reactions to FEIBA or any if its components, including factors of the kinin generating system. • Disseminated intravascular coagulation (DIC). • Acute thrombosis or embolism (including myocardial infarction). WARNINGS AND PRECAUTIONS Thromboembolic Events Thromboembolic events (including venous thrombosis, pulmonary embolism, myocardial infarction, and stroke) can occur with FEIBA, particularly following the administration of high doses (above 200 units per kg per day) and/or in patients with thrombotic risk factors [see ADVERSE REACTIONS]. Patients with DIC, advanced atherosclerotic disease, crush injury, septicemia, or concomitant treatment with recombinant factor VIIa have an increased risk of developing thrombotic events due to circulating tissue factor or predisposing coagulopathy. Potential benefit of treatment with FEIBA should be weighed against the potential risk of these thromboembolic events. Monitor patients receiving more than 100 units per kg of body weight of FEIBA for the development of DIC, acute coronary ischemia and signs and symptoms of other thromboembolic events. If clinical signs or symptoms occur, such as chest pain or pressure, shortness of breath, altered consciousness, vision, or speech, limb or abdomen swelling and/or pain, discontinue the infusion and initiate appropriate diagnostic and therapeutic measures. Hypersensitivity Reactions Hypersensitivity and allergic reactions, including severe anaphylactoid reactions, can occur following the infusion of FEIBA. The symptoms include urticaria, angioedema, gastrointestinal manifestations, bronchospasm, and hypotension. These reactions can be severe and systemic (e.g., anaphylaxis with urticaria and angioedema, bronchospasm, and circulatory shock). Other infusion reactions, such as chills, pyrexia, and hypertension have also been reported. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of FEIBA and provide appropriate supportive care. Transmission of Infectious Agents Because FEIBA is made from human plasma it may carry a risk of transmitting infectious agents, e.g., viruses, and the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. The risk has been minimized by screening plasma donors for prior exposure to certain viruses, by testing for the presence of certain current virus infections and by inactivating and removing certain viruses during the manufacturing process [see DESCRIPTION in full Prescribing Information]. Despite these measures, the product may still potentially transmit human pathogenic agents. There is also the possibility that unknown infectious agents may still be present. All infections thought by a physician to have been possibly transmitted by this product should be reported by the physician or other healthcare providers to Baxter Healthcare Corporation, at 1-800-423-2862 (in the U.S.) and /or to FDA Med Watch (1-800-FDA1088 or www.fda.gov/medwatch). ADVERSE REACTIONS The most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting.

Baxalta and Feiba are trademarks of Baxalta Incorporated April 2016 USBS/145/16-0015

The serious adverse reactions seen with FEIBA are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis. Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice. The safety assessment of FEIBA is based on the review of the data from two prospective clinical trials in which FEIBA was used for the treatment of acute bleeding episodes and a prospective trial that compared the use of FEIBA prophylactically versus on-demand treatment. The adverse reactions reported from two prospective clinical trials in which FEIBA was used for the treatment of acute bleeding episodes were chills, chest pain, chest discomfort, dizziness, dysgeusia, dyspnea, hypoesthesia, increase of inhibitor titer (anamnestic response), nausea, pyrexia, and somnolence. Specifically, the first trial was a multicenter randomized, double-blind trial in 15 hemophilia A subjects with inhibitors to factors VIII. The second trial was a multicenter FEIBA study conducted in 44 hemophilia A subjects with inhibitors, 3 hemophilia B subjects with inhibitors and 2 acquired factor VIII inhibitor subjects. Of the 489 infusions used to treat acute bleeds during the second trial, 18 (3.7%) caused minor transient reactions of chills, fever, nausea, dizziness and dysgeusia. Out of 49 subjects, 10 (20%) had a rise in their inhibitor titers after treatment with FEIBA. Five of these subjects (50%) had increases that were, tenfold or more, and 3 (30%) of these subjects received factor VIII or IX concentrates within 2 weeks prior to treatment with FEIBA. These anamnestic rises were not associated with decreased efficacy of FEIBA. Table 2 lists the adverse reactions in >5% of subject reported in the randomized, prospective prophylaxis trial comparing FEIBA prophylaxis with on-demand treatment in 36 hemophilia A and B subjects with inhibitors to factors VIII or IX. The trial population included 33 (92%) subjects with hemophilia A and 3 (8.3%) subjects with hemophilia B. Four (11%) subjects were ≥7 to <12 years of age, 5 (14%) were ≥12 to <16 years of age, and 27 (75%) were ≥16 years of age. A total of 29 (80.6%) subjects were Caucasian, 3 (8.3%) Asian, 2 (5.6%) Black/African American, and 2 (5.6%) other. The subjects received a total of 4,513 infusions (3,131 for prophylaxis and 1,382 for ondemand). Adverse reactions were defined as adverse events that occurred (a) within 24 hours after being infused or (b) adverse events assessed related or possibly related or (c) adverse events for which the investigator’s or sponsor’s opinion of causality was missing or indeterminate. Table 2 Prophylaxis Study Adverse Reactions (ARs) in >5% of Subjects MedDRA System Organ Class Blood And Lymphatic System Disorders Gastrointestinal Disorders Investigations Musculoskeletal And Connective Tissue Disorders

Preferred Term

Number Number of of ARs Subjects

Percent of Subjects (N=36)

Anemia

5.6

Diarrhea Nausea Vomiting Hepatitis B Surface Antibody Positive

2 2 2

5.6 5.6 5.6

11.1

Hemarthrosis

8.3

Post-Marketing Experience Because post-marketing reporting of adverse reactions is voluntarily and from a population of uncertain size, it is not always possible to reliably estimate the frequency of these reactions or establish a causal relationship to product exposure. BLOOD AND LYMPHATIC SYSTEM DISORDERS: disseminated intravascular coagulation CARDIAC DISORDERS: tachycardia, flushing RESPIRATORY, THORACIC, AND MEDIASTINAL DISORDERS: bronchospasm, wheezing GASTROINTESTINAL DISORDERS: abdominal discomfort SKIN AND SUBCUTANEOUS TISSUE DISORDERS: pruritus GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS: malaise, feeling hot, injection site pain DRUG INTERACTIONS Concomitant Medications Consider the possibility of thrombotic events when systemic antifibrinolytics such as tranexamic acid and aminocaproic acid are used during treatment with FEIBA. No adequate and wellcontrolled studies of the combined or sequential use of FEIBA and recombinant factor VIIa or antifibrinolytics have been conducted. Use of antifibrinolytics within approximately 6 to 12 hours after the administration of FEIBA is not recommended.

Integrity, Accuracy, Empathy...

FEATURE

8 I Medical Cannabis: The Safer Solution To Chronic Pain? For those who are skeptical, or “the anti-cannabis type”, this is a must-read. Dr. Sulak explains the true benefits and risks of medicinal cannabis, while giving you a few things you may not have considered. First, read then draw your own conclusions. For all of our readers, we hope you learn something new.

CONTENTS COMMUNITY CHATTER 5 I Inhibitor Family Camp at the Painted Turtle: Milestones This spring marked the launch of CHES’ 7th year of Inhibitor Family Camp. Carri Nease and her family attended the program 5 years ago for the first time, marking the beginning of a tradition. Carri explains why they keep coming back.

6 I HFA’s 2016 Annual Symposium: Forecasting Resilience Kelly Pintarelli (mother of a child with inhibitors) points out the theme of HFA’s 2016 Annual Symposium, “Together, We are Resilient!” as she attended the program with her husband and son. She explains how HFA stayed true to that statement.

BLOODLINES 26 I A New Factor 8 in Town: Weekly SUB-Q Treatment for FVIII’s with/without INHIBITORS Is ACE910 the miracle drug that could end the burdens of inhibitors? A bold statement, and probably not one that Roche cares to make (at least not yet.) But it could be a reality. Take a look at these results to decide for yourself!

FUN & INSPIRATION 27 I TopTEN: You Know You Have Joint Disease When...

WHAT’S NEW

Not moving like you used to? Use these top-ten signs to diagnose your joint disease (for entertainment purposes only).

20 I More Resources: For Those with an Inhibitor

WHAT’S the PLAN?

Learn about two new programs that might be helpful. HFA’s new Helping Hands now fills some of the gaps that were lost from Novo Nordisk’s transition from SevenSecureTM to NovoSecureTM, and meet Hope for Hemophilia, a new organization determined to offer support and assistance.

FAMILY MATTERS

32 I The Art of Transitioning With over 20 years of experience in the world of special education, and mother of three (two of which have hemophilia and/or inhibitors) Janet Brewer, M.Ed offers her guidance through four life stages to help you and your child make those necessary transitions a little smoother.

22 I Words are Powerful: So How About Watching Your Language It’s all too often a natural reaction to take a pessimistic stance on negative experiences. If you look at that experience... you may find that using positive and encouraging words could dramatically alter the outcome of your whole day. Try it!

LIFELINES for HEALTH

Spring 2016

CONTENTS

COMING SOON! CHES is presenting three new programs in 2016 for the bleeding disorder community. After the ShockTM is specially created for newly diagnosed families with an inhibitor ages 0-6 years. Visit aftertheshock.org for more info.

The LadyBugs ProgramTM is just for women (18 years and up) who are mothers, caregivers, and siblings of someone with a bleeding disorder, as well as women with bleeding disorders. Check ladybugsprogram.org for more info on these regional, one-day programs. The final program will be specific to offering education, self-advocacy, psychosocial support, and more for those with Glanzmann’s Thrombasthenia, a rare platelet disorder. Subscribe to our notifications for more info on this developing program at comphealthed.com/comm/. LifeLines for HealthSM Disclaimers The views and opinions of our writers are not a reflection of Comprehensive Health Education ServicesTM, Inc. (CHES), or its sponsors. This newsletter is designed to provide a forum for community members to express their views from an open and honest platform. It is meant to provide a sharing of knowledge and experience to help one another. Nothing in this newsletter is meant to replace the advice of your HTC, medical professional team or insurance provider. You are always urged to seek the opinion of a healthcare professional for treatment and your specific insurance provider for information. We take your privacy very seriously. We would never disclose your personal health information without your express written consent. We would never sell nor make available our secure database to anyone. Articles and pictures may not be reproduced, published, and/or placed on websites without the express written permission of CHES. In every publication of LifeLines for HealthSM, we will provide links to other websites that are not owned or controlled by CHES or its sponsors. We cannot be responsible for privacy practices of other website owners, nor can we be responsible for the accuracy of the information provided.

Letter From the Editors At CHES, we are proud to say that we have nearly doubled the educational programming we have to offer those in the smaller sub pockets of the bleeding disorder community. We currently have three programs for those with an inhibitor: Inhibitor Family CampTM (IFC) is offered twice per year, on the west coast in the spring at The Painted Turtle and in the fall on the east coast at Victory Junction. IFC is for families with a child with an inhibitor ages 6-18 that provides a combined educational and recreational experience at camp. Over 55% of our IFC campers report that their child cannot attend regular hemophilia camp for a variety of reasons. IFC provides a unique blend of activities for all ages in a safe, fun environment. After (the) ShockTM, is a new regional program for families with a child ages 0-6 who has been newly diagnosed with an inhibitor. LeverageTM, is an innovative outdoor education program for adults with an inhibitor ages 18 and up. GutMonkey has partnered with us to bring this perceived high-risk adventure to give those with an inhibitor the opportunity to break down some of the barriers they may see in their lives. Activities such as high ropes and rappelling assist participants to see that barriers can be overcome with the help of teamwork and modifications. All of our programs are supported by competitive, educational grants from manufacturers to provide these experiences free of charge to our families. CHES is an independent, educational, privately owned company run by two members of the hemophilia and inhibitor community who care passionately about our families. As we often say, “we don’t just know inhibitors, with live them.” This issue’s Feature shines a light on the topic of medical marijuana. Twenty-four states have legalized it for medical use, while California, Colorado, Oregon, Washington and the District of Columbia have now made it legal for recreational use. Multiple states are reevaluating their stance on marijuana use for both recreational and medicinal purposes. In my little town of Hanson, Massachusetts we are #2 out 10 of the highest number of opioid overdoses here on the South Shore. Multiple states are struggling with this crisis. Perhaps marijuana could be the “gateway” drug to help stop opioid addiction? In our Community Chatter section, as always we bring you news of events and programs specific to the inhibitor community written by community members. We look forward to our writer’s perspective in every edition, and we love to share their perspective about programming for our community. Our BloodLines section turns its attention to the new factor in town that is creating a lot of buzz in the inhibitor community. Roche’s ACE910 (Emicizumab) promises weekly subcutaneous treatment for Factor VIII patients with or without inhibitors. This could truly be a life changing product for so many. What’s New (formally the Insurance Corner) provides several resources available to the hemophilia and inhibitor community. HFATM in response to the changes made in other assistance programs created an additional category to their Helping Hands program to specifically assist families with an inhibitor. We also shine a light on Hope for Hemophilia, a non-profit organized in 2009 that provides critical support for so many of us that are often one hospitalization away from financial disaster. Do you or your family live with inhibitors or suffer with inhibitors? That very subtle language change can make a huge difference in how you view your life. In Family Matters, Dr. McClain reminds us to watch our language. Words like awful, worst, terrible-do they allow you to view the situation as it is, or make you feel like a victim? The words we use can have a powerful effect on how we react to situations we are often faced with when dealing with a chronic illness. I think I am going to put this article on my refrigerator. We often think about transitioning in the hemophilia community as that time when our teens start to take on their care in preparation for their 18th birthday. In What’s the Plan, if we think about transitioning beginning the moment we encourage our children to prep the infusion site with an alcohol pad, the road to transition isn’t just a short period of time. It becomes a road where you are always coaching your child to become adult advocates for themselves. After all, you won’t be going to college with them, will you? Fun and Inspiration is meant to provide the levity and triumphs we need as we navigate this sometimes-bumpy path called our lives. We hope that this summer gives you the opportunity to relax and rejuvenate. Find 10 minutes for yourself everyday to affirm that you are an amazing strong resilient human being! Do you have an idea, comment or suggestion? We really want to hear from you! - Janet Brewer & Eric Lowe “Cheerfulness is the best promoter of health and is as friendly to mind as to the body.” -Joseph Addison jbrewer@comphealthed.com l elowe@comphealthed.com

Inhibitor Family Camp at the Painted Turtle

Milestones

Written by: Carri Nease

ur family first attended Inhibitor Family Camp in 2012 when the boys were six (6) after patiently waited since we preregistered them at the age of four (4)! We didn’t know what to expect, but we were all very excited.

a bit.

It felt good knowing that for the most part, camp Tyin a fter h was set up for pretty much is 2 nd succe ssful anything we could throw at self-in fusion them on a normal day with hemophilia & W e After a LONG Trip, we arrived at The inhibitors. even managed Painted Turtle. Shawn & I went on a walk together a couple milestones with the This would be the first weekend we had around the camp, just holding hands- it help of nurses Kathy Byrne and Brianna O’Neil (who are members of CHES’ ever walked away from our boys and not was glorious. traveling infusion nursing team) and The worried about them (much). The camp Inhibitor Family Camp has only gotten pairs a family with a volunteer family better over the years since our first arrival. Painted Turtle’s Well Shell staff. Tyin was able to infuse himself for the second pal who provides a second pair of hands Comprehensive Health Education time and Connor worked through his keeping track of the kids so that the parents can have breathing room to attend Services, with an education grant fear to get nearly to the point of his first sessions while the kids can run off and supported by Novo Nordisk, increased the stick! He actually managed to hold the camp weekend by one day, which needle and touch where he would have participate in LOTS of activities. allowed us to actually recover some infused. This is a huge step for someone from the travel and enjoy the feel of with a deep needle phobia. the festivities more. Connor also performed with a camp This year, we weren’t sure if we buddy in the talent show - another huge would be able to attend. Tyin was milestone! having a particularly rough time Registration was a bit of a challenge but and we weren’t sure if he would it is always worth it. be healthy enough to make the As a parent attending Inhibitor Family trip. He worked hard though, and Camp, I get the strength and support I physical therapy cleared him so need, both from the other parents AND we could make it. from the CHES staff and The Painted Turtle in 2012 Tyin walked and played more al to the Painted riv ar st Turtle staff including the Well Shell Staff. fir e’s as The Ne this weekend than he had in the past several months because of the Camp was teaching us it was okay to activities and encouragement of friends. let go and let someone else take over for We had so much fun! continued on pg 31

LIFELINES for HEALTH

Spring 2016

COMMUNITY CHATTER

HFA’s 2016 Annual Symposium:

Forecasting Resilience

by Kelly Pintarelli

ogether, We are Resilient!! This was the theme of HFA’s 2016 Symposia in Las Vegas, Nevada. It was the first year that Bubba and I had the opportunity to attend a HFA Symposium, and it was truly an amazing experience. Although, we have attended prior Inhibitor Summits and Inhibitor Family Camp, I was excited to see the whole bleeding disorder community come together at one event.

The stymposium venue in Las Vegas was beautiful, and I was very thankful HFA chose a resort that was away from all of the traffic and excitement on the Vegas Strip. It provided an atmosphere where 1,300 members of the bleeding community could come together to learn, teach, support, share and enjoy what I like to call “The Hemophilia Normal.” Even though there were a large number of us who face that daily that I did not recognize, it became readily apparent from day one that these people were already my family. An adopted family of amazing individuals that already understood sleepless nights with devastating bleeds, missed birthday parties, unfulfilled work and school commitments, endless mobility issues, ports, PICC lines, infections, long hospital admissions, anxiety, PTSD, grief and loss. My heart was full!!!

The educational track that HFA put together was outstanding! They did a wonderful job of providing sessions that keep us informed of what is new and upcoming in the world of hemophilia. This included a glance at where we are and where we have come from, current research programs, how to navigate insurance, financial finesse, aging with a bleeding disorder, how to raise resilient children, and how to be your own best advocate. We also had a chance to talk about insurance trends, woman bleeders, social media, and had several relevant rap sessions. HFA also planned a week of entertaining activities for both the younger and older kids. Bubba loved being able to participate in yoga, martial arts and the children’s art project. I loved hearing about the adventures he had and friendships he had made during the day. My personal favorite part of HFA (besides the amazing pool and weather) was the full, one-day, educational track dedicated to families with inhibitors. This was a track that was thoughtfully put together by a committee of individuals from the bleeding community and included direct input from individuals living with inhibitors. It has been my experience over the last 9 years that having an inhibitor is a huge game changer when living with hemophilia. Bubba was diagnosed at 10 months with a high titer inhibitor and it truly has been a difficult journey. So, I was very excited about this track to say the least! This year’s inhibitor track really dug into the “meat” of inhibitors. This was not a track for individuals that needed inhibitors 101. It truly gave caregivers and family members the chance to dive deeper into living with an inhibitor and issues that surround it.

LIFELINES for HEALTH

Spring 2016

It included information on the new inhibitor program through Helping Hands, a journey through inhibitors with inhibitor patients, sessions on joint health, emotional health, state level advocacy and what is on the horizon for inhibitors. The most resonating session during the inhibitor track for me was Two Roads Diverged: Patient Journeys with Inhibitors. During this session we were able to listen to a very candid conversation from 4 inhibitor patients ranging from teenager to adult. They spoke about their journey with inhibitors, what has worked, and what has not. I was so deeply touched by this session and found myself tearing up while re-living with each of them both their struggles and their triumphs. It was a very cathartic experience for me. It showed me that even if Bubba never tolerates (and he may not) that he can still have a very long , experiential, fulfilling life. It also reiterated that life with inhibitors does not exist in a straight line. There will continue to be a lot of trials and tribulations and what is most important is how you respond to them. What works for one family may not work for another -and that is okay. Overall I would say that HFA 2016 was an amazing experience. The knowledge gained from others in the community and educational sessions was truly invaluable. I am definitely excited to attend next yearâ&#x20AC;&#x2122;s HFA Symposium in Rhode Island.

COMMUNITY CHATTER

Medical Medical Cannabis Cannabis The The Safer Safer Solution Solution To To Chronic Chronic Pain Pain

hronic pain and opiate addiction have ruined countless lives and changed the landscape of medicine.

Some patients feel let down by the medical system when they are denied effective treatment for chronic pain. Others feel injured by medical providers who have started them on opiate medications that have led to addiction and ruined their lives. Healthcare providers feel frustrated that they cannot do more to safely help patients with chronic pain, guilty for providing effective short-term treatments with harmful longterm effects, and depleted of compassion for patients who need help.

By Dr Dustin Sulak D.O.

Plus, patients can grow their own. The vast majority of medical cannabis patients use this herbal medicine to treat chronic pain.

Luckily, there is another solution that is safe, effective, and sustainable.

The saf

LIFELINES for HEALTH

Spring 2016

FEATURE

The providers at my medical practice are amazed by the results of medical cannabis in the treatment of chronic pain. Patient after patient tells a similar story, and it goes something like this: “I cut my opiate usage drastically, I’m sleeping better, feeling happy, and enjoying my life

more, all with less side effects.” We find that whenever we see a patient who has been using opiates to treat chronic pain, after starting medical cannabis

the opiate dosage decreases by at least 50% in the first week. It’s often more

like a 60-80% reduction. Many patients go on to completely discontinue opiate medications over the next several months. They often reduce or discontinue other pain medications as well, including antidepressants and anti-seizure drugs.

fer solution to chronic pai

FEATURE

Scientific research tells the same story. A review article published in Life Sciences (2004) concludes, “The administration of low doses THC in conjunction with low doses of morphine seems to be an alternative regimen that reduces the need to escalate opioid dose while increasing opioid potency.” An animal study found that adding a small amount of THC increased the effectiveness of morphine by four to twelve times. And while cannabinoids, like THC, can work together with opioids to increase potency in the pain centers of

the brain, there isn’t a significant effect on the brainstem, the area responsible for decreased heart rate, slower breathing, and ultimately death in the case of an opiate overdose. This means that using cannabis with opioids is safer than using opioids alone. Patients are starting to figure this out. A study published in the Harm Reduction Journal (2009) looked at 350 medical cannabis users, and found that 65% of them were using cannabis as a substitute for prescription drugs, 40% were using it as a substitute for alcohol, and 26% were using it as a substitute for illicit drugs. Three of the subjects used cannabis to help them quit smoking tobacco. The most common reasons for the substitution were less adverse side effects, better symptom management, and less withdrawal. So while we all were told that cannabis is dangerous because it is a “gateway drug,” scientists are now calling it an, “exit drug” within the framework of harm reduction. The goal is to substitute a safer drug for one that’s more dangerous in patients who are unable to achieve total abstinence.

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Spring 2016

And cannabis is certainly safer than most of our current options for treating chronic pain. In 1999, the Institute of Medicine published a report that stated, “Except for the harms associated with smoking, the adverse effects of cannabis use are within the range tolerated for other medications,” and “there is no

conclusive evidence that marijuana causes cancer in humans.”

Unlike opiate medications, cannabis can remain effective at a low dose for years or decades. While opiates can numb the pain and disconnect the mind from the body, increasing risk for inappropriate activity and re-injury, cannabis often does the opposite. Patients report that after using cannabis, the pain is still there but is less intense, less bothersome, and they no longer have to dwell on it. They often feel more connected to their bodies because they no longer have to retreat from the pain. Patients feel able to reclaim their lives from the constant focus on pain. Cannabis does not have to be smoked, as many patients are using tinctures (liquid extracts) and vaporizers (smoke-free inhalation). Certified patients are able to grow their own or purchase the medicine from caregivers or dispensaries.

Choosing the Right Medicine Strains There are hundreds of varieties (i.e. strains, cultivars) of medical cannabis, each with different medicinal effects. The unique properties of each strain arise from their synergy of the cannabinoids, terpenes, and other compounds. Finding the right medicine will likely require trying out a number of varieties, but the following guidelines can help direct your search. Most patients eventually find 2-3 different strains that work well for different purposes, such as one for daytime and one for sleep. Medical marijuana strains fall into a few major categories: indica, sativa, hybrid indica/sativa, and high-CBD. While these terms are not scientifically accurate, they are widely used in the cannabis world. Furthermore, cannabis by one name from one producer may be vastly different than cannabis by the same name from a different producer. The best way to identify a strain that works or doesn’t work for you is to have it laboratory tested for cannabinoid and terpene content, and to remember the appearance and aroma infusing fresh buds.

Choose one: 0 CANNABIS C STAVIA 0 CANNABIS INDICA 0 HYBRID INDICA/SATIVA 0 CBD-RICH

FEATURE

CANNABIS SATIVA strains are tall plants with narrow leaves and buds often having a spicy or flowery fragrance. These varieties are generally more mentally stimulating, more energetic and euphoric. They tend to help more with nausea and other abdominal complaints, appetite stimulation, headaches, depression and fatigue. Although side effects are uncommon and usually mild, sativa strains are more likely to cause or worsen anxiety or paranoia.

cannabis lotions, salves or patches to the skin. Each delivery method will have a different medicinal effect, even when using the same strain of cannabis. There is no “best” delivery method – each has it’s benefits, drawbacks, and clinical utility in certain situations.

CANNABIS INDICA strains are shorter and bushier plants and often having sour or fruity fragrance. Indica varieties are typically relaxing and sedating. They tend to help more with pain, anxiety, muscle spasms, and insomnia.

Inhalation of cannabis has several advantages, including fast onset, ease in consuming the correct dosage, and convenience. It’s an ideal delivery for patients with nausea, vomiting, or other conditions that make swallowing difficult. Due to the rapid onset of inhaled cannabis, it’s easiest for users to judge their response and know how much the cannabis is helping and whether or not they need more. On the other hand, inhaled cannabis is more likely to cause cardiovascular side effects, has a shorter duration than other methods, and may have a higher abuse potential.

HYBRID INDICA/SATIVA strains are very common, and have been bred to achieve specific qualities. Many hybrid strains are able to produce effects that provide the best of both worlds. CBD-RICH strains have specifically been bred to produce high levels of cannabidiol (CBD). CBD has several exciting medicinal properties, including anti-anxiety, anti-inflammatory, anti-tumor, anti-seizure, and pain relief. Strains with high levels of CBD have decreased our negligible psychoactivity, making them desirable for patients who want the medical benefits without getting “high” or impaired. This can be ideal for patients who need to drive, work, etc. We recommend you consult with an experienced healthcare provider to receive personalized recommendations on cannabis strains and cannabinoid ratios/dosages for your condition. If you cannot find an expert, you can network with other cannabis-using patients that have similar conditions and find out which strains work the best for them, but remember, unless you purchase from the same producer, you may end up with totally different product.

Cannabis does not have to be smoked. The beneficial compounds in cannabis can be administered by inhaling smoke or vapors, ingesting cannabis added to food or drink, swallowing capsules, taking liquid extracts such as tinctures or oils absorbed through the mouth or swallowed, or applying

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INHALATION: ONSET 1-5 MINUTES, DURATION 1-6 HOURS

Smoking is perhaps the most traditional method of cannabis use, and while it works well for many patients, it has some disadvantages. The heat and smoke irritate the respiratory tract and lungs and could potentially worsen conditions such as asthma and COPD. While the smoke itself contains carcinogenic (cancer-causing) substances, long term heavy marijuana

smokers do not have an increased risk of lung cancer, likely due to

the anti-cancer properties of cannabis offsetting the carcinogens in the smoke. Herbal vaporizers are very popular with many medical marijuana patients, and are widely considered to be the healthiest way to inhale cannabis. Studies have shown vaporizers reduce the amount of harmful substances created compared to burning and smoking. The vaporizer heats the herb to a specific temperature between 300 and 400 degrees Fahrenheit, releasing the medicinal substances into a vapor without the plant material burning. The vapor produced is warm and nonirritating. It contains the smell and flavor of the cannabis but feels as if you are breathing air. A wide variety of vaporizers can be found on the market today, and many require some practice before achieving optimal use. When a patient uses a vaporizer it may take a little longer for the therapeutic effects of the cannabis to be felt compared to smoking. Many novices make the initial mistake of taking too many puffs in their first use, thinking they are not getting the medicine because it does not feel like smoke in their lungs. You can avoid this mistake by following our Introduction to Cannabis and Cannabis Sensitization programs. TOPICAL (EXTERNAL USE ON SKIN): ONSET AND DURATION VARIABLE Cannabis or its oil-based extracts can be added to balms, lotions, salves, patches, and linaments. Many of these preparations can be made at home and applied topically to help alleviate pain, muscle spasms, inflammation, itching, and various skin conditions, including

eczema. Topical use of cannabis typically does not produce psychoactive effects if used in small amounts and in small areas. Using large amounts over significant areas of the body can cause psychoactive effects. Topicals labeled “transdermals” are designed for better absorption and will have a full body effect. The efficacy of a topical preparation may depend on its potency, but many patients have found they can use flower/bud trimmings or leaves to produce an effective topical medicine. LIQUID CANNABIS EXTRACTS: ONSET 10-45 MINUTES, DURATION 2-8 HOURS A tincture is a liquid extraction of cannabis, often in a solution of alcohol. Sometimes the word tincture is erroneously used to describe oil infusions, which can be used in a similar manner. Liquid extracts can be absorbed directly through the mucous membrane in the mouth for faster onset, or they can be swallowed. This is one of the most versatile delivery methods and works well for most patients. They are convenient, discreet (minimal odor), and easy to dose correctly. The onset and duration are intermediate compared to ingestion or inhalation. Liquid medicines do have some challenges: active constituents can settle to the bottom of the bottle, the effects are variable if held in the mouth vs swallowed, alcohol tinctures can irritate the mouth and may be inappropriate for people with a history of alcohol abuse, and the taste can be bitter or spicy. To use a liquid cannabis extract, a specific number of drops or milliliters can be placed under the tongue and held for 1-2 minutes before swallowing. Brushing one’s teeth first can increase blood flow in the mouth and speed the onset. Because each batch of liquid medicine may have a different potency, be sure to look at laboratory testing results, or if unavailable, start each new batch with a few drops and increase slowly. When using an alcohol based tincture, add the liquid to a small amount of warm or hot water first – this will

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evaporate some of the alcohol and dilute the rest, preventing irritation to your mouth. INGESTION: ONSET WITHIN 1-2 HOURS, DURATION 4-12 HOURS Cannabis can be added to a wide range of capsules, foods, and beverages. When cannabis is swallowed, it is absorbed through the GI tract and then metabolized in the liver (known as first-pass metabolism), which changes the cannabinoids into a form with longer duration of action and somewhat different medical properties. Many users report ingestion produces a stronger psychoactive effect, while others report less psychoactivity, and most find that ingestion produces somewhat different therapeutic effects. Many patients find that once they know their optimal dose, ingestion is a superior delivery method due to convenience, long duration, and preferred effects. Ingestion can be complicated by erratic absorption from one day to the next â&#x20AC;&#x201C; absorption may depend on other foods consumed, sleep, stress, and other factors that affect the motility of the gastrointestinal tract. Also, cannabis edibles may not have a homogenous distribution of cannabinoids throughout the entire product â&#x20AC;&#x201C; one half of the cookie may be more potent than the other half. It is often difficult to achieve the correct dosage when consuming cannabis edibles, mostly because it takes so long for the patient to know if he or she has taken too little or too much. For these reasons, we emphasize caution when eating cannabis; it is a good idea to start with a very small amount, wait 2-3 hours, then repeat the dose if needed. While it is not dangerous to overdose on edible cannabis, the effects can be quite unpleasant, sometimes aggravating the symptoms a patient

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wishes to improve. Since so many patients make dosage mistakes with cannabis-infused foods and have unpleasant experiences, we recommend keeping novice cannabis users avoid edibles and use cannabis in the form of a medicine, not a food. Please note that while raw cannabinoids have nonpsychoactive therapeutic properties, the effects commonly associated with cannabis are due to cannabinoids that have been heated (decarboxylated). Before being consumed, cannabis must be heated to convert the cannabinoids into their active form. RECTAL (Variable onset and duration) Cannabis suppositories can be useful for treating conditions in the pelvis and low back, and in patients who are unable or prefer not to inhale or swallow cannabis. Some patients report that rectal administration is less-psychoactive than eating, which may be due to the avoidance of first-pass liver metabolism. The little research on rectal cannabis absorption shows wide variability in absorption, and the composition of the suppository can make a significant difference. RAW CANNABIS PREPARATIONS (ONSET 2-45 MINUTES, DURATION 2-8 HOURS) Almost all of the research on the therapeutic properties of cannabinoids has examined the compounds after they have been exposed to heat, an activation process known as decarboxylation. A small body of literature has shown that raw cannabinoids possess anti-inflammatory and antinausea properties at surprisingly low doses in rodents. Patients have reported that raw cannabinoids are also effective for treating pain and seizures.

Raw cannabis is non-psychoactive and has a higher content of terpenes, the substances that produce the aroma, which also have a broad range of therapeutic effects. While very large doses can cause some digestive upset, few other side effects have been reported from raw cannabis. Fresh raw cannabis flowers can be eaten directly (chewing for a few minutes can speed onset), prepared into an oil, or brewed into tea (boil 1g cannabis in 1 L of water for 15 minutes). This recipe of cannabis tea produces a beverage with 90% raw cannabinoids and 10% decarboxylated, and quickly loses its potency over the course of a week, even when stored in the refrigerator.

Resources Brenneisen, R., et al. “The effect of orally and rectally administered delta 9-tetrahydrocannabinol on spasticity: a pilot study with 2 patients.” International journal of clinical pharmacology and therapeutics 34.10 (1996): 446-452. Earleywine, Mitch, and Sara Smucker Barnwell. “Decreased respiratory symptoms in cannabis users who vaporize.” Harm Reduct J 4.11 (2007). Elsohly, Mahmoud A., et al. “Rectal bioavailability of delta9-tetrahydrocannabinol from various esters.” Pharmacology Biochemistry and Behavior 40.3 (1991): 497-502. Gieringer, Dale, Joseph St. Laurent, and Scott Goodrich. “Cannabis vaporizer combines efficient delivery of THC with effective suppression of pyrolytic compounds.” Journal of Cannabis Therapeutics 4.1 (2004): 7-27. Grotenhermen, Franjo. “Pharmacokinetics and pharmacodynamics of cannabinoids.” Clinical pharmacokinetics 42.4 (2003): 327-360. Hart, Carl L., et al. “Comparison of smoked marijuana and oral Δ9-tetrahydrocannabinol in humans.”Psychopharmacology 164.4 (2002): 407-415. Hazekamp, Arno, et al. “Cannabis tea revisited: A systematic evaluation of the cannabinoid composition of cannabis tea.” Journal of ethnopharmacology 113.1 (2007): 85-90. Rock, E. M., et al. “Tetrahydrocannabinolic acid reduces nausea‐induced conditioned gaping in rats and vomiting in Suncus murinus.” British journal of pharmacology 170.3 (2013): 641-648.

Photo courtesy of Melissa Mullen Photography

About Dr Sulak: Dr Dustin Sulak, D.O. is a renowned integrative medicine physician based in Maine, whose practice balances the principles of osteopathy, mind-body medicine and medical cannabis. Regarded as an expert on medical cannabis nationally, Dr. Sulak educates medical providers and patients on its clinical use, while continuing to explore the therapeutic potential of this ancient yet emerging medicine. He received undergraduate degrees in nutrition science and biology from Indiana University, a doctorate of osteopathy from the Arizona College of Osteopathic Medicine, and completed an internship at MaineDartmouth Family Medicine Residency. Material for this article was originally posted on www.Healer.com written by Dr. Dustin Sulak and may be viewed here http://healer.com/

Verhoeckx, Kitty CM, et al. “Unheated Cannabis sativa extracts and its major compound THC-acid have potential immunomodulating properties not mediated by CB 1 and CB 2 receptor coupled pathways.”International immunopharmacology 6.4 (2006): 656-665.

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TAYLOR, JACK, BOB, HENRY, SID, JUSTIN, CHASE, AND KALI (Clockwise from top right) Taylor has congenital FVII deficiency; Jack, Bob, Sid, Justin, and Chase have congenital hemophilia with inhibitors; Kali has Glanzmann’s thrombasthenia

Rooted in effective bleed control Reaching to help more patient types than any other factor product • 30 years of research and long-term clinical experience has gone into NovoSeven® RT – Compassionate use, also known as expanded access, began in 1988 – FDA approval received in 1999 • NovoSeven® RT is used to treat more patient types than any other factor product – Treatment of bleeding and prevention of bleeding for surgeries and procedures in adults and children with: – Hemophilia A or B with inhibitors – Congenital factor VII deficiency – Glanzmann’s thrombasthenia with a decreased or absent response to platelet transfusions – Treatment of bleeding and prevention of bleeding for surgeries and procedures in adults with acquired hemophilia • More than 70 trials and registries completed, with a commitment to ongoing research

Visit NovoSevenRT.com today to learn more.

Indications and Usage NovoSeven® RT (Coagulation Factor VIIa [Recombinant]) is used for: • Treatment of bleeding and prevention of bleeding for surgeries and procedures in adults and children with hemophilia A or B with inhibitors, congenital Factor VII (FVII) deﬁciency, and people with Glanzmann’s thrombasthenia who have a decreased or absent response to platelet transfusions • Treatment of bleeding and prevention of bleeding for surgeries and procedures in adults with acquired hemophilia

Important Safety Information WARNING: BLOOD CLOTS • Serious blood clots that form in veins and arteries with the use of NovoSeven® RT have been reported. • Your healthcare provider should discuss the risks and explain the signs and symptoms of blood clots to you. Some signs of a blood clot may include pain, swelling, warmth, redness, or a lump in your legs or arms, chest pain, shortness of breath, or sudden severe headache and/or loss of consciousness or function. • Your healthcare provider should monitor you for blood clots during treatment with NovoSeven® RT. Warnings and Precautions • NovoSeven® RT should be used with caution if you have an increased risk for blood clots, such as with disseminated intravascular coagulation (DIC), clogged arteries, crush injury, septicemia (an infection in the blood), uncontrolled bleeding after giving birth, history of heart disease, liver disease, limited movement following surgery, in elderly people, in newborns, or if you are taking aPCCs/PCCs (activated or nonactivated prothrombin complex concentrates) with NovoSeven® RT. • Allergic reactions, including serious whole body allergic reactions, have been reported with NovoSeven® RT. Tell your healthcare provider if you are allergic to NovoSeven® RT, any of its ingredients, or mice, hamsters, or cows. If you think you are having an allergic reaction, call your healthcare provider right away. Some signs of allergic reaction may include shortness of breath, rash, itching, redness of the skin, and fainting/dizziness. • People with Factor VII deﬁciency should be monitored by their healthcare provider for antibodies, which can cause NovoSeven® RT to stop working properly. Side Effects • The most common and serious side effects are blood clots. • Tell your healthcare provider about any side effects that bother you or do not go away. Use with Other Drugs • Blood clots may occur if NovoSeven® RT is given with Coagulation Factor XIII (13). You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. NovoSeven® RT is a prescription medicine. Novo Nordisk provides patient assistance for those who qualify. Please call 1-877-NOVO-777 (1-877-668-6777) to learn more about Novo Nordisk assistance programs.

Please see Brief Summary of Prescribing Information on the following pages.

Novo Nordisk Inc., 800 Scudders Mill Road, Plainsboro, New Jersey 08536 U.S.A. NovoSeven® is a registered trademark of Novo Nordisk Health Care AG. Novo Nordisk is a registered trademark of Novo Nordisk A/S. © 2015 Novo Nordisk All rights reserved. 0715-00027616-1 September 2015

NovoSeven ® RT Coagulation Factor VIIa (Recombinant) Rx only BRIEF SUMMARY. Please consult package insert for full prescribing information. WARNING: THROMBOSIS: Serious arterial and venous thrombotic events following administration of NovoSeven ® RT have been reported. Discuss the risks and explain the signs and symptoms of thrombotic and thromboembolic events to patients who will receive NovoSeven ® RT. Monitor patients for signs or symptoms of activation of the coagulation system and for thrombosis. [See Warnings and Precautions] INDICATIONS AND USAGE: NovoSeven® RT (Coagulation Factor VIIa [Recombinant]) is a coagulation factor indicated for: Treatment of bleeding episodes and peri-operative management in adults and children with hemophilia A or B with inhibitors, congenital Factor VII (FVII) deficiency, and Glanzmann’s thrombasthenia with refractoriness to platelet transfusions, with or without antibodies to platelets; Treatment of bleeding episodes and peri-operative management in adults with acquired hemophilia CONTRAINDICATIONS: None known. WARNINGS AND PRECAUTIONS: Thrombosis: Serious arterial and venous thrombotic events have been reported in clinical trials and postmarketing surveillance. Patients with disseminated intravascular coagulation (DIC), advanced atherosclerotic disease, crush injury, septicemia, or concomitant treatment with aPCCs/PCCs (activated or nonactivated prothrombin complex concentrates) and uncontrolled post-partum hemorrhage have an increased risk of developing thromboembolic events due to circulating tissue factor (TF) or predisposing coagulopathy [See Adverse Reactions and Drug Interactions]. Exercise caution when administering NovoSeven ® RT to patients with an increased risk of thromboembolic complications. These include, but are not limited to, patients with a history of coronary heart disease, liver disease, disseminated intravascular coagulation, postoperative immobilization, elderly patients and neonates. In each of these situations, the potential benefit of treatment with NovoSeven® RT should be weighed against the risk of these complications. Monitor patients who receive NovoSeven ® RT for development of signs or symptoms of activation of the coagulation system or thrombosis. When there is laboratory confirmation of intravascular coagulation or presence of clinical thrombosis, reduce the dose of NovoSeven ® RT or stop the treatment, depending on the patient’s condition. Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis have been reported with NovoSeven® RT. Administer NovoSeven ® RT only if clearly needed in patients with known hypersensitivity to NovoSeven ® RT or any of its components, or in patients with known hypersensitivity to mouse, hamster, or bovine proteins. Should symptoms occur, discontinue NovoSeven ® RT, administer appropriate treatment and weigh the benefit/risks prior to restarting treatment with NovoSeven ® RT. Antibody Formation in Factor VII Deficient Patients: Factor VII deficient patients should be monitored for prothrombin time (PT) and factor VII coagulant activity before and after administration of NovoSeven ® RT. If the factor VIIa activity fails to reach the expected level, or prothrombin time is not corrected, or bleeding is not controlled after treatment with the recommended doses, antibody formation may be suspected and analysis for antibodies should be performed. Laboratory Tests: Laboratory coagulation parameters (PT/INR, aPTT, FVII:C) have shown no direct correlation to achieving hemostasis. Assays of prothrombin time (PT/INR), activated partial thromboplastin time (aPTT), and plasma FVII clotting activity (FVII:C), may give different results with different reagents. Treatment with NovoSeven ® has been shown to produce the following characteristics: PT: As shown below, in patients with hemophilia A/B with inhibitors, the PT shortened to about a 7-second plateau at a FVII:C level of approximately 5 units per mL. For FVII:C levels > 5 units per mL, there is no further change in PT. The clinical relevance of prothrombin time shortening following NovoSeven ® RT administration is unknown. PT (sec) PT versus FVII:C INR: NovoSeven® has demonstrated the ability to 14 normalize INR. However, INR 13 values have not been shown 12 to directly predict bleeding 11 outcomes, nor has it been 10 possible to demonstrate the 9 impact of NovoSeven® on 8 bleeding times/volume in 7 models of clinically-induced 6 bleeding in healthy volunteers who had received Warfarin, 5 when laboratory parameters 4 (PT/INR, aPTT, thromboelas3 togram) have normalized. 2 aPTT: While administration of 1 NovoSeven® shortens the 0 30 40 prolonged aPTT in hemo0 10 20 philia A/B patients with FVII:C (unit per mL)

inhibitors, normalization has usually not been observed in doses shown to induce clinical improvement. Data indicate that clinical improvement was associated with a shortening of aPTT of 15 to 20 seconds. FVIIa:C: FVIIa:C levels were measured two hours after NovoSeven ® administration of 35 micrograms per kg body weight and 90 micrograms per kg body weight following two days of dosing at two hour intervals. Average steady state levels were 11 and 28 units per mL for the two dose levels, respectively. ADVERSE REACTIONS: The most common and serious adverse reactions in clinical trials are thrombotic events. Thrombotic adverse reactions following the administration of NovoSeven ® in clinical trials occurred in 4% of patients with acquired hemophilia and 0.2% of bleeding episodes in patients with congenital hemophilia. Clinical Trials Experience: Because clinical studies are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug product cannot be directly compared to rates in clinical trials of another drug, and may not reflect rates observed in practice. Adverse reactions outlined below have been reported from clinical trials and data collected in registries. Hemophilia A or B Patients with Inhibitors: In two studies for hemophilia A or B patients with inhibitors treated for bleeding episodes (N=298), adverse reactions were reported in ≥2% of the patients that were treated with NovoSeven® for 1,939 bleeding episodes (see Table below). Table: Adverse Reactions Reported in ≥2% of the 298 Patients with Hemophilia A or B with Inhibitors Body System Reactions Body as a whole Fever Platelets, Bleeding, and Clotting Fibrinogen plasma decreased Cardiovascular Hypertension

# of adverse reactions (n=1,939 treatments)

(n=298 patients)

# of patients

Serious adverse reactions included thrombosis, pain, thrombophlebitis deep, pulmonary embolism, decreased therapeutic response, cerebrovascular disorder, angina pectoris, DIC, anaphylactic shock and abnormal hepatic function. The serious adverse reactions of DIC and therapeutic response decreased had a fatal outcome. There have been no confirmed reports of inhibitory antibodies against NovoSeven ® or FVII in patients with congenital hemophilia A or B with alloantibodies. In two clinical trials evaluating safety and efficacy of NovoSeven ® administration in the perioperative setting in hemophilia A or B patients with inhibitors (N=51), the following serious adverse reactions were reported: acute post-operative hemarthrosis (n=1), internal jugular thrombosis adverse reaction (n=1), decreased therapeutic response (n=4) Congenital Factor VII Deficiency: Data collected from the compassionate/ emergency use programs, the published literature, a pharmacokinetics study, and the Hemophilia and Thrombosis Research Society (HTRS) registry showed that 75 patients with Factor VII deficiency had received NovoSeven® : 70 patients for 124 bleeding episodes, surgeries, or prophylaxis; 5 patients in the pharmacokinetics trial. The following adverse reactions were reported: intracranial hypertension (n=1), IgG antibody against rFVIIa and FVII (n=1), localized phlebitis (n=1). As with all therapeutic proteins, there is a potential for immunogenicity. Patients with factor VII deficiency treated with NovoSeven ® RT should be monitored for factor VII antibodies. The incidence of antibody formation is dependent on the sensitivity and specificity of the assay. Additionally, the observed incidence of antibody (including neutralizing antibody) positivity in an assay may be influenced by several factors including assay methodology, sample handling, timing of sample collection, concomitant medications, and underlying disease. For these reasons, comparison of the incidence of antibodies to NovoSeven ® RT with the incidence of antibodies to other products may be misleading. Acquired Hemophilia: Data collected from four compassionate use programs, the HTRS registry, and the published literature showed that 139 patients with acquired hemophilia received NovoSeven ® for 204 bleeding episodes, surgeries and traumatic injuries. Of these 139 patients, 6 patients experienced 8 serious adverse reactions. Serious adverse reactions included shock (n=1), cerebrovascular accident (n=1) and thromboembolic events (n=6) which included cerebral artery occlusion, cerebral ischemia, angina pectoris, myocardial infarction, pulmonary embolism and deep vein thrombosis. Three of the serious adverse reactions had a fatal outcome. Glanzmann’s Thrombasthenia: Data collected from the Glanzmann’s Thrombasthenia Registry (GTR) and the HTRS registry showed that 140 patients with Glanzmann’s thrombasthenia received NovoSeven® RT for 518 bleeding episodes, surgeries or traumatic injuries. The following adverse reactions were reported: deep vein thrombosis (n=1), headache (n=2), fever (n=2), nausea (n=1), and dyspnea (n=1). Postmarketing Experience: The following adverse reactions have been identified during post approval use of NovoSeven®. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship.

Table: Post Marketing Experience MedDRA System Preferred Term Organ Class Immune system Hypersensitivity (including anaphylactic shock, disorders flushing, urticaria, rash, angioedema) Vascular disorders Thromboembolic events (including hepatic artery thrombosis, myocardial infarction, cerebral infarction, intestinal infarction, intracardiac thrombus, peripheral ischemia, portal vein thrombosis, myocardial ischemia, renal artery thrombosis) DRUG INTERACTIONS: Avoid simultaneous use of activated prothrombin complex concentrates or prothrombin complex concentrates. The risk of a potential interaction between NovoSeven ® RT and coagulation factor concentrates has not been adequately evaluated in preclinical or clinical studies. Do not mix NovoSeven ® RT with infusion solutions. Thrombosis may occur if NovoSeven ® RT is administered concomitantly with Coagulation Factor XIII. USE IN SPECIFIC POPULATIONS: Pregnancy: Pregnancy Category C. There are no adequate and well-controlled studies in pregnant women. NovoSeven ® RT should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Treatment of rats and rabbits with NovoSeven ® in reproduction studies has been associated with mortality at doses up to 6 mg per kg body weight and 5 mg per kg body weight respectively. At 6 mg per kg body weight in rats, the abortion rate was 0 out of 25 litters; in rabbits at 5 mg per kg body weight, the abortion rate was 2 out of 25 litters. Twenty-three out of 25 female rats given 6 mg per kg body weight of NovoSeven® gave birth successfully, however, two of the 23 litters died during the early period of lactation. No evidence of teratogenicity was observed after dosing with NovoSeven ®. Labor and Delivery: There are no adequate and well-controlled studies in labor, delivery, and postpartum periods. NovoSeven® RT has caused thrombosis when used to control post-partum hemorrhage. Nursing Mothers: It is not known whether NovoSeven ® RT is excreted in human milk. Because many drugs are excreted in human milk, and because of the potential for serious adverse reactions in nursing infants, a decision should be made whether to discontinue nursing or to discontinue the drug, taking into account the importance of the drug to the mother. Pediatric Use: Clinical trials enrolling pediatric patients were conducted with dosing determined according to body weight and not according to age. Hemophilia A or B with Inhibitors: During the investigational phase of product development NovoSeven ® was used in 16 children aged 0 to <2 years for 151 bleeding episodes, 27 children aged 2 to <6 years for 140 bleeding episodes, 43 children aged 6 to <12 for 375 bleeding episodes and 30 children aged 12 to 16 years for 446 bleeding episodes. In a double-blind, randomized comparison trial of two dose levels of NovoSeven ® in the treatment of joint, muscle and mucocutaneous hemorrhages in hemophilia A and B patients with and without inhibitors 20 children aged 0 to <12 and 8 children aged 12 to 16 were treated with NovoSeven ® in doses of 35 or 70 micrograms per kg dose. Treatment was assessed as effective (definite relief of pain/tenderness as reported by the patient and/or a measureable decrease of the size of the hemorrhage and/or arrest of bleeding within 8 hours [rated as excellent = 51%], within 8-14 hours [rated as effective = 18%] or after 14 hours [rated as partially effective = 25%]) in 94% of the patients. NovoSeven® was used in two trials in surgery. In a dose comparison 22 children aged 0 to 16 years were treated with NovoSeven ®. Effective intraoperative hemostasis (defined as bleeding that had stopped completely or had decreased substantially [rated as effective = 86%] or bleeding that was reduced but continued [rated as partially effective = 9%]) was achieved in 21/22 (95%) patients. Effective hemostasis was achieved in 10/10 (100%) patients in the 90 mcg/kg dose group and 10/12 (83%) in the 35 mcg/kg dose group at 48 hours; effective hemostasis was achieved in 10/10 (100%) in the 90 mcg/kg dose group and 9/12 (75%) in the 35 mcg/kg dose group at 5 days. In the surgery trial comparing bolus (BI) and continuous infusion (CI) 6 children aged 10 to 15 years participated, 3 in each group. Both regimens were 100% effective (defined as bleeding has stopped completely, or decreased substantially) intra-operatively, through the first 24 hours and at day 5. At the end of the study period (Postoperative day 10 or discontinuation of therapy) hemostasis in two patients in the BI group was rated effective and hemostasis in one patient was rated as ineffective (defined as bleeding is the same or has worsened). Hemostasis in all three patients in the CI group was rated as effective. Adverse drug reactions in pediatric patients were similar to those previously reported in clinical trials with NovoSeven ®, including one thrombotic event in a 4 year old with internal jugular vein thrombosis after porta-cath placement which resolved. Congenital Factor VII deficiency: In published literature, compassionate use trials and registries on use of NovoSeven ® in congenital Factor VII deficiency, NovoSeven ® was used in 24 children aged 0 to <12 years and 7 children aged 12 to 16 years for 38 bleeding episodes, 16 surgeries and 8 prophylaxis regimens. Treatment was effective in 95% of bleeding episodes (5% not rated) and 100% of surgeries. No thrombotic events were reported. A seven-month old exposed to NovoSeven® and various plasma products developed antibodies against FVII and rFVIIa [see Clinical Trials Experience and Overdosage]. Glanzmann’s Thrombasthenia: In the Glanzmann’s Thrombasthenia Registry, NovoSeven ® was used in 43 children aged 0 to 12 years for 157 bleeding episodes and in 15 children aged 0 to 12 years for 19 surgical procedures. NovoSeven® was also used in 8 children aged >12 to 16 years for 17 bleeding episodes and in 3 children aged >12 to

16 years for 3 surgical procedures. Efficacy of regimens including NovoSeven ® was evaluated by independent adjudicators as 93.6% and 100% for bleeding episodes in children aged 0 to 12 years and >12 to 16 years, respectively. Efficacy in surgical procedures was evaluated as 100% for all surgical procedures in children aged 0 to 16 years. No adverse reactions were reported in Glanzmann’s thrombasthenia children. Geriatric Use: Clinical studies of NovoSeven ® RT in congenital factor deficiencies and Glanzmann’s thrombasthenia did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. OVERDOSAGE: Dose limiting toxicities of NovoSeven ® RT have not been investigated in clinical trials. The following are examples of accidental overdose. One newborn female with congenital factor VII deficiency was administered an overdose of NovoSeven ® (single dose: 800 micrograms per kg body weight). Following additional administration of NovoSeven ® and various plasma products, antibodies against rFVIIa were detected, but no thrombotic complications were reported. One Factor VII deficient male (83 years of age, 111.1 kg) received two doses of 324 micrograms per kg body weight (10-20 times the recommended dose) and experienced a thrombotic event (occipital stroke). One hemophilia B patient (16 years of age, 68 kg) received a single dose of 352 micrograms per kg body weight and one hemophilia A patient (2 years of age, 14.6 kg) received doses ranging from 246 micrograms per kg body weight to 986 micrograms per kg body weight on five consecutive days. There were no reported complications in either case.

More detailed information is available upon request. For information contact: Novo Nordisk Inc. 800 Scudders Mill Road Plainsboro, NJ 08536, USA 1-877-NOVO-777 www.NovoSevenRT.com Manufactured by: Novo Nordisk A/S 2880 Bagsvaerd, Denmark License Number: 1261 Novo Nordisk® is a registered trademark of Novo Nordisk A/S. NovoSeven® is a registered trademark of Novo Nordisk Health Care AG. © 2015 Novo Nordisk 0715-00027826-1 8/15

Resources

by Janet Brewer, M.Ed

For Those with An Inhibitor

or many of us in the inhibitor community, financial support when needed isn’t something nice to have, but something critical to have. Our rent, utility bills, and car payments don’t stop when we are in the hospital for weeks to months on end. One parent-caregiver must always be with a child (or the adult with an inhibitor) resulting in a loss of much needed income. We rely on understanding employers and personal time off to maintain job security and income. For many however, those two important pieces may not be a reality resulting in a sea of debt that is difficult to recover from.

In December 2015, the SevenSecureTM program sponsored by Novo Nordisk changed to the NovoSecureTM program creating a loss of much needed support for inhibitor patients. Many of us relied on their assistance for bills not covered by insurance, subsidies to attend meetings to gain a better understanding of life with an inhibitor, and more.

HFA Helping Hands Program

t any point, families living with a bleeding disorder can Families affected by inhibitors are faced with unique medical experience financial hardship because of their medical and financial needs. The inhibitor community is challenged situation. The Helping Hands Program has become a safety with bleed management, joint damage, venous access connet for hundreds of families throughout the years. Helping cerns, pain issues, and social and emotional distress. In addiHands has provided many families with assistance for extion to all these is a great financial burden. In 2016, Helping On January 4, 2016 HFA filled that gap, bypenses including inhibitors to transportation. their well-established Helping Hands Program to include: like housing, utilities, and In addition Hands expanded to provide support for families affected by 1 Emergency Assistance, Items Reimbursement, Inhibitor Resources. to assistance for Support these basic and livingFinancial expenses, Helping Hands inhibitors through the addition of Inhibitor Support. has helped individuals and families to obtain durable medical items for the care of their bleeding disorders.

Hemophilia Federation of America - Helping Hands

•

Emergency Assistance: supports families with urgent funding for basic living expenses such as housing, transportation, and utility bills Items Reimbursement: reimburses individuals with the cost of durable medical equipment and other medically necessary items Inhibitor Support: supports families affected by inhibitors with the cost of educational travel, medical transportation, and tutoring/educational supplies. Financial Resources: provides assistance for those in the bleeding disorder community who are uninsured, under insured, or experiencing lapses in insurance coverage. It also offers assistance to help families facing financial strain get access to factor product.

The following chart provides a visual of what is included. For further enrollment and registration please navigate to: http://www.hemophiliafed. org/programs/helping-hands/.

Use the Following Chart to Navigate the Coverage Helping Hands Provides: What is covered?

How often?

Who can apply?

LIFELINES for HEALTH

Items Reimbursement

Inhibitor Support

Basic living expenses:

Durable medical items

Educational travel,

housing, utilities,

(i.e., medical IDs, protective

medical travel,

transportation

gear, braces, cooling/heating

tutoring/educational

supplies, fitness support)

supplies

Throughout the year,

depending on needs/

requests

Once per year

Individuals and families

with a diagnosed bleeding

with a diagnosed

disorder

bleeding disorder and an inhibitor with measurable titer and/or shortened half life

Referral

Referral from eligible referrer*

Referral from eligible

referrer and/or self-referral

*Please reach out to our Helping Hands team for additional information regarding eligible referrer.

1. Resources: Hemophilia Federation of America: Programs and Services-Helping Hands

Emergency Assistance

Spring 2016

Hope for Hemophilia While at HFA, I had the pleasure of meeting Rachel Neyland, Patient Care Coordinator at Hope for Hemophilia, a non-profit established in 2009 by Jonathan and Carla James. Their mission is to be a conduit of hope, strength and resources to individuals and their families through seasons of crisis caused by hemophilia. They provide two programs for families affected by hemophilia: The Patient Resource program offers education for families regarding resources such as Helping Hands, PSI, co-payment and manufacturer co-pay and compassionate use programs. They also provide information regarding scholarships available within the hemophilia community. Hope for Hemophilia assists families to navigate through federal, state and local programs such as the United Way, Social Security Disability, as well as local food banks and community charities. The Direct Financial Assistance program supplies financial assistance for those in the hemophilia community who are in crisis. A crisis is categorized into one of three tiers: • • •

Crisis One: an emergency caused by surgery due to bleeds, loss of job and/or income caused by excessive bleeding episodes or injuries that necessitate a long recovery Crisis Two: a general financial crisis that includes job loss and income due to complications caused by hemophilia for a child Crisis Three: a personal financial need due to monetary strain

Tier one crises are funded on a first-come first-serve basis. Tier two and three are provided as funds become available.

Hope for Hemophilia provides up to $1000.00 per household per year. Funds are distributed directly to the debtor and no money is provided directly to the family or individual. To find out more, refer to http://hopeforhemophilia.com

WHAT’S NEW?

Words are

POWERFUL So How About Watching Your Language? by Dr. Gary McClain, PhD

LIFELINES for HEALTH

Spring 2016

hink back to the last time you had a really bad day. What was going on that day to make it so bad? And now, think about the words you might have used when you described it. Did you really let loose with a big old rant on how bad things were? Maybe. “I hate this.” “This is the worst.” “It’s just awful.”

Take a Step Back and Listen to Yourself So, the first question to ask yourself: Was it really all that hateful, awful, the worst? Now, think about how those words may have impacted the way you were feeling that day. Were you feeling optimistic that whatever it was you were dealing with, it wasn’t something you hadn’t been through before and would get through this time too? Or were you feeling like the world might just be crashing down around you, and that tomorrow might be even worse? With words like “hate,” “worst,” and “awful,” chances are you weren’t feeling all that optimistic.

FAMILY MATTERS

So here’s what I’m getting at: There’s something kind of magical about words. The reason I say that words are magical is because the words we use have a very big impact on how we experience the world around us. For better, or for not so better. I’m talking not only about the words you say to other people, but the words you say to yourself. Think about the last time things didn’t go the way you wanted them to go. You know, one of those times that seem to come along all too often when you are living with a bleeding disorder.

And if you are a family member of someone living with a bleeding disorder, think about the words you might be using when your loved one hits a rough spot. Think back to the words you used to describe what happened. On one hand, you might have talked in a way that implied that, while it was disappointing, it was at least something you could figure out a way to cope with, or even live with. Or you might have instead talked about it as if it was the worst thing that could possibly happen, soon to result in misery and despair. Now, think about how the way you talked about the situation made you feel. Most likely, if you talked about it terms of possibilities, you felt disappointed/frustrated/annoyed, but not without options. But if you talked about it with words like “worst” and “awful,” you may have felt hopeless and defeated. Take it a step further. The language you use when you talk about the challenges that come your way also affects the actions you take, or don’t take. If you tell yourself that a situation is the absolute worst thing imaginable, you are setting yourself up to view it as so insurmountable that you may as well not even try to expect anything better. And so you stay miserable and stuck.

Words are powerful! The words we say to ourselves and the words we say to others. When you are first diagnosed with a chronic condition, and every experience is new, those words of desperation and defeat are more likely to be top of mind. But now it’s also the time to be aware of the words you are using, and try to use words that encourage you to stay optimistic. In other words, watch your language. Here are some of my favorite words to watch out for: The “hate” word. There’s not much to love or even like about the symptoms of a chronic condition or the medication regimen you may be learning to live with, along with the lifestyle changes. But do you hate it? Hate opens up the door to negativity, making everything a punishment when, if you look at it objectively, it doesn’t have to be. Look at it this way: How can you hate something that is helping to make you feel better?

LIFELINES for HEALTH

Spring 2016

“Living with” or “suffering from?” This is a big one. If you’re living with a bleeding disorder, chances are you learned to face life on life’s terms, to do what you need to take the best possible care of yourself. Empowered. When you are suffering from your chronic condition, you place yourself in the role of victim, with things being done to you that you have no control over. You have a choice. “Awful.” Using the word awful serves to put a situation in such a negative light that a set-back can feel like the world is crashing around you. That’s called “awfulizing.” Ask yourself: Is it really awful? Or is it just inconvenient? Or disappointing? Or frustrating? How you answer those questions will make a big difference in terms of the potential solutions you are able to identify. “The worst.” This word goes hand-inhand with “awful.” With the same impact on your outlook. “Impossible” or “never.” Now, here’s a way to stress yourself out. Allow your mind to jump to the worst (there’s that word again) possible scenario and follow it down the rabbit hole. One of the best ways to conjure up this scenario is through the use of “impossible” or “never.” Because if a situation truly is impossible, if things will never get any better, why even try? Both of these words leave you backed into a corner with no way out. Again, as the victim. And you don’t have to be.

Talk Optimism, Stay Optimistic Here’s how to keep your language more positive: Choose to focus on what’s working.

Not only on what’s not working. Remind yourself of what’s good in your life. Be grateful. Say it to yourself and say it out loud. Remind yourself: I’ve been through this before. If you’re living with a bleeding disorder, or with someone who is, then this is probably not the first time you’ve felt this way. It might help to remember that you’re not in uncharted territory, and that you can get through it again.

and frustrations. Reach out to someone who can listen without judgment and give you some fresh perspective. Sure, living with a bleeding disorder brings surprises and responsibilities that you didn’t plan for. And yes, you’re not in control of everything that comes your way. But the words you use can create optimism or defeat – in your mind and in your actions. So, as your mom used to warn you: Watch your language!

Review your foundation. You’ve got a lot going for you. A health care team. Support. Knowledge. Coping skills. You’re resilient, right? Get support. When you’re having a hard time, it’s all too easy to kind of shut down and get trapped in your own fears

Gary McClain, PhD, LMHC, is a therapist, patient advocate, and author, specializing in helping clients deal with the emotional impact of chronic and life-threatening illnesses, as well as their families and professional caregivers. He works with them to understand and cope with their emotions, to learn about their lifestyle and treatment options, to maintain compliance with medical regimens, to communicate effectively with the medical establishment, and to listen to their own inner voice as they make decisions about the future. His email is: gary@JustGotDiagnosed.com. He welcomes your questions and comments.

FAMILY MATTERS

A New Factor 8 in Town

by Stephen Brewer, B.Sc.

Roche’s ACE910 (Emicizumab) promises weekly SUBCUTANEOUS treatment for Factor VIII patients with or without INHIBITORS

oche Pharmaceuticals announced the breakthrough therapy designation granted by the US FDA in September 2015 for their novel treatment for patients twelve years or older with Hemophilia A with an inhibitor, termed ACE910 (pronounced /ās-nain-ten/).1 The approval was granted following two Phase I studies, the first in patients without hemophilia,1 and the second in patients with hemophilia A with and without inhibitors.2

ACE910 is described as a recombinant molecule that mimics the function of Factor VIII (FVIII), designed under the rationale of avoiding the complications due to developing or an already developed inhibitor, as well as improving on route and frequency of administration.3 Preliminary studies in the Phase I human trial in both non-inhibitor and inhibitor patients investigated weekly subcutaneous prophylactic treatment, resulting in an overall reduction in median annualized bleed rates (ABR) in all patients.2

(BU/mL). Two patients were escalated from the 0.3 to 1.0 mg/kg dosage group as a result of frequent bleeding.

Results

ACE910 is preliminarily shown to be safe and effective in long-term study, decreasing the median annualized bleed rate (ABR) in all three-dosage groups, irrespective of inhibitors.

Procedure The phase I study involved eighteen Japanese patients, ages 1258, with severe hemophilia A with and without inhibitors, receiving a once-weekly subcutaneous dose for a total of twelve weeks, with median follow-up of nine months for sixteen out of eighteen patients.2,4 Three dosage groups, of 0.3, 1.0 and 3.0 mg/kg were observed, with both inhibitor and non-inhibitor patients in each dosage group, with inhibitors ranged from 3-111 Bethesda Units

The median ABR was reduced from: •

• •

32.5 to 2.0 bleeds per year for the lowest dosage group6 18.3 to 1.2 for the moderate dose group,6 and

15.2 to 0.0 for the highest dose group6

ACE910, when dosed weekly, effectively brought patients with severe hemophilia with or without inhibitors to annualized bleed rates similar to that of

moderate hemophiliacs5,2 with no adverse events indicative of hypercoagulation even in patients treating bleeds on demand with FVIII or bypassing agents.2 It should be noted that while no antibodies to ACE910 were developed during the initial twelve week study, in an extended phase I/II study involving sixteen of the eighteen original patients, two patients were observed to have developed anti-ACE910 antibodies, but did not seem to affect ACE910’s efficacy according to the investigators.6

This information is limited and preliminary; the current Phase III study that launched in November 2015 is set to complete in January of 2018. The Phase III study is set globally, and features prophylaxis over a twenty-four week period with an estimated seventy patients. More information about the current Phase III study can be found below under the drug name Emicizumab. ID Number: BH29884 NCT Identifier: NCT02622321

Full Source of Clinical Trial Data: https://clinicaltrials.gov/show/ NCT02622321

1 Roche Investor Update. US FDA grants breakthrough therapy designation for Roche’s investigational medicine ACE910 for people with haemophilia A with factor VIII inhibitors. Available at: http://www.roche.com/investors /updates/inv-update-2015-09-04.htm. Last accessed March 2016. 2 Shima M. et al. Dec. 2014. Safety and Prophylactic Efficacy Profiles of ACE910, a Humanized Bispecific Antibody Mimicking the FVIII Cofactor Function, in Japanese Hemophilia A Patients Both without and with FVIII Inhibitors: First-in-Patient Phase 1 Study. Blood: 124(21):691.

Uchida N. et al. Dec. 2015. A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects. Blood: 127 (13).

Shima M. et al. 2015. Long-term safety and prophylactic efficacy of once-weekly subcutaneous administration of ACE910, in Japanese hemophilia A patients with and without FVIII inhibitors: interim results of the extension study of a phase 1 study. 4

Den Uijl I. et al. 2014. Outcome in moderate haemophilia. Blood Transfus; 12 Suppl 1: s330-6.

LIFELINES for HEALTH

Spring 2016

BLOODLINES

YOU Have

You

Know

Arthritis

by Eric Lowe

When...

’ll be turning 36 this year. So to some, I’m still a baby, and perhaps to others, I’m already over the hill. I don’t feel old. And as a matter of fact, I often feel like a teenager wandering aimlessly through the guantlet of life. But my joints (who have lived with inhibitors for the last 34 years) tell a different story. If they could talk, they would say, “sit down, you’re old!” But I am still young in spirit, so I refuse to stop moving. And I hope that all of you out there share the same mindset. But in the meantime, let’s have a little fun with it. For those who are wondering if you have arthritis, grab a pencil and check off all that apply.

10. 0

Your body has more snap, crackle, pops than Rice Krispies.

The carpet padding in your home has more layers than an onion.

You can’t sneak through the house without disturbing others because it sounds like you’re stepping on bubble wrap, and…

Someone meets you for the first time and they think your limp is a pimp-walk.

That’s when you realize your lifelong dream of becoming a stealthy ninja is over.

Instead of working out, you layer clothing to “bulk up”.

You pretend you’re reading the movie credits while standing at your seat, but you’re really just loosening up your joints for the long walk to the car.

Your joints have more rough spots than Selena Gomez and Justin Bieber.

The Eiffel Tower has more movement than your joints.

You can forecast rain better than your local meteorologist.

How’d you do? If you checked: 1-3: Keep truckin’ away, junior. You’ve got a lot more miles to go. But don’t plan on winning any races unless you own a scooter. 4-7: You’re certified to play the sympathy card. See how many body massages you can get in a week. And work on your pout-pout face in the mirror to increase your numbers. 8-10: You’re ready for retirement. And you might want to donate your body to science. They’re looking for global phenomenoms like youself. And if you specifically checked off #4, congratulations. You’re still a kid at heart too!

For entertainment purposes only. See full disclaimer on page 4.

LIFELINES for HEALTH

Spring 2016

FUN & INSPIRATION

NOW AVAILABLE

INDICATIONS KOVALTRY® is a medicine used to replace clotting factor (Factor VIII or antihemophilic factor) that is missing in people with hemophilia A. KOVALTRY® is used to treat and control bleeding in adults and children with hemophilia A. KOVALTRY® can reduce the number of bleeding episodes in adults and children with hemophilia A when used regularly (prophylaxis). Your healthcare provider may give you KOVALTRY® when you have surgery. KOVALTRY® is not used to treat von Willebrand Disease.

To learn more about KOVALTRY®, connect with your local Bayer Representative at KOVALTRYexpert.com.

IMPORTANT SAFETY INFORMATION You should not use KOVALTRY® if you are allergic to rodents (like mice and hamsters) or any ingredients in KOVALTRY®. Tell your healthcare provider if you have heart disease or are at risk for heart disease. The common side effects of KOVALTRY® are headache, fever, and itchy rash.

Don’t let insurance or financial challenges get in between you and your treatment

(Bayer Ad) Trial Program at no cost to you Assistance during gaps in insurance coverage Co-pay support Patient support programs Live Helpline Support

CALL 1-800-288-8374 NOW! 8:00 AM-8:00 PM (ET) Monday-Friday.

Spanish-speaking Case Specialists are also available.

Restrictions apply. Please visit KOVALTRY.com or call 1-800-288-8374 for more information about the restrictions. Talk to your doctor to see if KOVALTRY® is right for you.

IMPORTANT SAFETY INFORMATION (CONT’D) Allergic reactions may occur with KOVALTRY®. Call your healthcare provider right away and stop treatment if you get tightness of the chest or throat, dizziness, decrease in blood pressure, and nausea. Your body can also make antibodies, called “inhibitors,” against KOVALTRY®, which may stop KOVALTRY® from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to Factor VIII. Tell your healthcare provider about any side effect that bothers you or that does not go away. Call your healthcare provider right away if bleeding is not controlled after using KOVALTRY®. For additional important risk and use information, please see the Brief Summary of the Prescribing Information on the following page. You are encouraged to report negative side effects or quality complaints of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. Bayer, the Bayer Cross, and KOVALTRY are registered trademarks of Bayer. © 2016 Bayer. All rights reserved. Printed in USA 03/16 PP-675-US-0170

HIGHLIGHTS OF FDA-Approved Patient Labeling Patient Information KOVALTRY (KOH-vahl-tree) Antihemophilic Factor (Recombinant) This leaflet summarizes important information about KOVALTRY with vial adapter. Please read it carefully before using this medicine. This information does not take the place of talking with your healthcare provider, and it does not include all of the important information about KOVALTRY. If you have any questions after reading this, ask your healthcare provider. Do not attempt to self-infuse unless you have been taught how by your healthcare provider or hemophilia center. What is KOVALTRY? KOVALTRY is a medicine used to replace clotting factor (Factor VIII or antihemophilic factor) that is missing in people with hemophilia A (also called “classic” hemophilia). Hemophilia A is an inherited bleeding disorder that prevents blood from clotting normally. KOVALTRY is used to treat and control bleeding in adults and children with hemophilia A. Your healthcare provider may give you KOVALTRY when you have surgery. KOVALTRY can reduce the number of bleeding episodes in adults and children with hemophilia A when used regularly (prophylaxis). KOVALTRY is not used to treat von Willebrand Disease. Who should not use KOVALTRY? You should not use KOVALTRY if you • are allergic to rodents (like mice and hamsters). • are allergic to any ingredients in KOVALTRY. What should I tell my healthcare provider before I use KOVALTRY? • Tell your healthcare provider about all of your medical conditions. • Tell your healthcare provider and pharmacist about all of the medicines you take, including all prescription and non-prescription medicines, such as over-the-counter medicines, supplements, or herbal remedies. • T ell your healthcare provider if you have been told you have heart disease or are at risk for heart disease. • Tell your healthcare provider if you have been told that you have inhibitors to Factor VIII (because KOVALTRY may not work for you). What are the possible side effects of KOVALTRY? The common side effects of KOVALTRY are headache, fever and itchy rash. Allergic reactions may occur with KOVALTRY. Call your healthcare provider right away and stop treatment if you get tightness of the chest or throat, dizziness, decrease in blood pressure, and nausea. Your body can also make antibodies, called “inhibitors,” against KOVALTRY, which may stop KOVALTRY from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to Factor VIII.

These are not all the possible side effects with KOVALTRY. You can ask your healthcare provider for information that is written for healthcare professionals. Tell your healthcare provider about any side effect that bothers you or that does not go away. How do I store KOVALTRY? Do not freeze KOVALTRY. Store KOVALTRY at +2°C to +8°C (36°F to 46°F) for up to 30 months from the date of manufacture. Within this period, KOVALTRY may be stored for a period of up to 12 months at temperatures up to +25°C or 77°F. Record the starting date of room temperature storage clearly on the unopened product carton. Once stored at room temperature, do not return the product to the refrigerator. The product then expires after storage at room temperature for 12 months, or after the expiration date on the product vial, whichever is earlier. Store vials in their original carton and protect them from extreme exposure to light. Administer reconstituted KOVALTRY as soon as possible. If not, store at room temperature for no longer than 3 hours. Throw away any unused KOVALTRY after the expiration date. Do not use reconstituted KOVALTRY if it is not clear. What else should I know about KOVALTRY and hemophilia A? Finding veins for injections may be difficult in young children. When frequent injections are required, your healthcare provider may propose to have a device surgically placed under the skin to facilitate access to the bloodstream. These devices may result in infections. Medicines are sometimes prescribed for purposes other than those listed here. Do not use KOVALTRY for a condition for which it is not prescribed. Do not share KOVALTRY with other people, even if they have the same symptoms that you have. This leaflet summarizes the most important information about KOVALTRY. If you would like more information, talk to your healthcare provider. You can ask your healthcare provider or pharmacist for information about KOVALTRY that was written for healthcare professionals. Resources at Bayer available to the patient: For Adverse Reaction Reporting, contact Bayer Medical Communications 1-888-84-BAYER (1-888-842-2937) To receive more product information, contact KOVALTRY Customer Service 1-888-606-3780 Bayer Reimbursement HELPline 1-800-288-8374 For more information, visit www.KOVALTRY-us.com Bayer HealthCare LLC Whippany, NJ 07981 USA U.S. License No. 8 6907600BS

an Innovative CHES Program

Everyone deserves a camp to call their own This program is specially designed to meet the needs and limitations of children with both hemophilia & inhibitors. Immediate family members are also invited because we understand that chronic illness affects the whole family. We play, learn, and grow while we build a stronger community. To register, applicants must have an active inhibitor, or a history of an inhibitor within the last 12 months, and are between the ages of 6-18. If you’re interested, please act now. Space is limited, and slots are filled on a first come, first-served basis for those who qualify. New families have first priority. Questions? Don’t hesitate to call at: 781.878.8561 Supported by an educational grant from

SPRING SESSION

Date: ENDED - Friday, April 15th - Monday, the 18th, 2016 Loction: The Painted Turtle, located in Lake Hughes, CA Registration Opened: Friday, January 8th, 2016

FALL SESSION

Date: Friday, Oct. 7th - Monday, Oct. 10th, 2016 Loction: Victory Junction, located in Randleman, NC Registration Opens: Friday, July 1st, 2016

Presented by

To learn more or register visit: InhibitorFamilyCamp.org

(concluded from page 5)

Inhibitor Family Camp is the one event we look forward to each year where the kids are able to hang with kids like themselves. It helps them to hear that they are not the only kids with hemophilia who sometimes get multiple infusions in a day to combat a bleed. Or get stuck every day in hopes of beating back inhibitors so they can have a regular childhood. Inhibitor Family Camp is the Great Equalizer w and we are eagerly looking forward to returning next year.

Connor on the mic at talent

night

Despite the fact that it’s only a few short days, I leave feeling renewed and ready to tackle another year.

Inhibitor Family Camp at The Painted Turtle provided by CHES is the only “nearly guaranteed” camp we can be sure to attend in a year - health permitting. Inhibitors, bleeds and complicated treatment protocols have made hemophilia camp through our local chapter something we can’t count on right now. We do sign them up in hopes they can attend- but last year only one of the boys could go because the other had a bleed. I was in the ER with a disappointed child the day the chapter flew to camp.

LIFELINES for HEALTH

Spring 2016

Tyin & Con

nor playing

a life-size

d game of

chess

COMMUNITY CHATTER

Artof TRANSIT The

he Webster definition of transition is (n) passage from one state, stage, subject, or place to another (v) a movement, development, or evolution from one form, stage, or style to another. In our lifetime, we transition multiple times as life is always in perpetual motion. Heraclitus the Greek philosopher said, The only thing that is constant is change. As families or individuals managing hemophilia with an inhibitor, we have become experts at transitioning. Our lives change in the blink of an eye. One

moment we are sitting quietly enjoying a cup of coffee and the next thing we know we are inpatient. When life is going along “smoothly” we are waiting for the other shoe to drop. The art of transitioning is an ongoing process. One that begins the moment our lives are affected by hemophilia.

Transitioning in a school setting does not happen all at once either. There are four critical stages within the educational transitioning process; entry at the early childhood to kindergarten level, then middle school, high school, and finally college and/ or employment. Each level has its own areas of concern and set of communication and skill development. Stage 1: BE TRUE TO YOUR SCHOOL

One of the most critical aspects of this transition stage is cultivating an open, honest and trusting relationship with the elementary team. Our children are leaving our safe, controlled home environment and moving into what I would call as a teacher, “organized chaos”. There are now many more children

LIFELINES for HEALTH

Spring 2016

surrounding our child, increasing the risk of injury. This is a very stressful transition for every parent and child (as well as the school system). The first step is to meet with the school and institute an individualized health care plan (IHCP) that establishes protocols for any possible scenario to keep our children safe. There will be a lot of communication between you and your child’s teacher and/or school nurse to solidify this relationship. Annual transitions as the child advances through the grades will require reinforcement of that relationship with your school system and teachers. Miscommunications and mistakes are to be expected at each end of the relationship. The vast majority of school systems, especially teachers want to work with you for the benefit of your child’s success; it is there job. Try to remember that your child is one of at least twenty to twenty-five students s/he is responsible for every day. Learning about hemophilia does not happen overnight; it is a gradual progression of skills. The more often you practice appropriate communication skills to collaborate with your child’s school system, the better your skills will become. Stage 2: STUCK IN THE MIDDLE WITH YOU

Middle school is the next critical stage within the transitioning process. The positive communication skills you have (hopefully) developed by now will lay the groundwork for this chapter of multiple teachers and subjects. If your child does not have a 504 plan going into middle school, they should, as they will now be learning from a team of teachers. There may also be new members of the team who will become the point person or central advocate for your child such as a guidance counselor or school nurse. You should request that your 13-14 year

TIONING old child be invited to any meetings that involve them; which is all of them. They need to begin developing and honing the skills necessary to communicate their own needs effectively with your coaching. Some teachers may be uncomfortable with this or indicate that it is not their policy but by calmly explaining that you are teaching your child skills for a lifetime of self-advocacy and transitions, they will understand. You can expect that there may be three or four meetings per year during this transition stage for parentteacher conferences. There will also be a minimum of two 504 meetings, one at the beginning of the year and one at the end to add, delete or modify accommodations. If your child has an IEP, there will be one annual meeting with your child’s team unless they are up for a re-evaluation that occurs every three years. During a reevaluation year, you can expect more than one meeting, as eligibility is determined. Parents have asked me if they need to attend all these meeting every year and my response is always “YES”. Yes, it is many meetings. Dependent upon the health of your child and consistent communication, you may rely upon the point person in your team to update you regarding progress or concerns if you cannot attend them all. Make it a point however, to attend the first and last semester/trimester parent-teacher conferences. These are crucial for the year’s success and setting your child up for success and expectations for transitioning into the next grade. Stage 3: THE TIDES ARE HIGH

This now leads us to the high school transition year. These next four years will move at lightening speed; if you thought the elementary or middle school years went fast, this period of time feels like you blinked and it is over. You and your child will now be negotiating with multiple

teachers, multiple semesters and multiple meetings. Communication at this stage can be very difficult between you and your child. Make friends with your child’s teachers, you will need them! Chocolate and gift cards go a long way. Handwritten notes are ALWAYS appreciated. Make it a point to get to know the athletic director or PE teacher if your child has not been excused from PE. This period of noncommunication that began in middle school with your child will likely become exasperating to negotiate. The end goal is age 18 at which point your child is now considered an adult. YEP! Trust me, they use their well-established communication skills to remind you of it frequently starting with the year they turn 17. If your child’s high school has not linked them in with your states’ vocational rehabilitative agency by the age of 16, be sure to make that connection. Don’t forget your child should still be attending meetings (which they may not like) and the school district is federally obligated to invite them. It is wise to start asking by the time they are 15 to begin this process. Dependent upon the state, the eligibility process can take up to a year, so start early. You will find more information at http://www2.ed.gov/ programs/rsabvrs/index.html. Here you can find facts regarding your individual state’s governance over the Rehabilitation Act of 1973 and what services you or your child may be afforded. Each state is awarded different amounts of funding from the federal government so resources vary from state to state https://rsa.ed.gov/ people.cfm. Stage 4: SCHOOL’S OUT

No matter whether your child decides on a college career, a safe trade, or a year or two of figuring it out, they will need to use their self-advocacy skills with college admissions, disability services, or a boss.

by Janet Brewer, M.Ed

Once they reach 18, parents can no longer be provided with information regarding your child so it is all up to them. They can fall back on their resource contact from vocational rehabilitation no matter what career route they choose. Vocational rehabilitation services can include assistance with obtaining a job, training and in some states college funding. It is well worth the effort to get in touch with them! Just an aside, as a mother who has done the high school/college “rodeo” three times, relax! There is already so much pressure put on young adults these days. Here in Massachusetts with many prestigious private colleges, the message often received is that you must attend a private four-year institution and sleep away from home in order to be successful. This path may be the right one for some children, but not for all. Student loan debt is at an all time high with young adults coming out of college with massive debt and no jobs to help pay it. It will most likely take your child more than 4 years to complete their degree depending upon their health, their level of interest and how many times they change their major.

Try to remember that they have lived a life that makes them wise beyond their years in some ways but very naïve in others. Each generation’s lifespan extends longer and longer into old age. In this country, we work hard for a long period of time where leisure and recreational activities are just now being recognized as important to our overall health. They will follow their own path. It will probably make us crazy as parents to watch the trails they go down and what trials they bring; but they were raised to be the best advocates for themselves and we have to trust we taught them well.

WHAT’S the PLAN?

89 E. Washington Street Hanson, MA 02341-1125

CHES Mission To Inspire awareness and selfreliance for patients with chronic health conditions, their families, and their communities.

Editors in Chief Janet Brewer, M.Ed Eric Lowe

Publication Designer Eric Lowe

Contributing Writers: Janet Brewer, M.Ed Stephen Brewer, B.Sc. Eric Lowe Dr. Gary McClain, PhD Carri Nease Kelly Pintarelli Dr. Dustin Sulak, D.O.

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